FDA Approves SKYTROFA® (Lonapegsomatropin-tcgd) for the Once-Weekly Treatment of Adults with Growth Hormone Deficiency
Ascendis Pharma (Nasdaq: ASND) has received FDA approval for SKYTROFA® (lonapegsomatropin-tcgd) for treating adult growth hormone deficiency (GHD). This marks a significant expansion from its 2021 pediatric GHD approval. SKYTROFA is administered once-weekly, offering a less burdensome alternative to traditional daily somatropin injections.
The approval was based on the foresiGHt Phase 3 trial, which demonstrated efficacy and safety compared to placebo and daily somatropin. This milestone aligns with Ascendis's Vision 2030 goal to become the leading endocrinology rare disease company, with plans to initiate additional trials for various conditions in Q4 2025.
Ascendis Pharma (Nasdaq: ASND) ha ottenuto l'approvazione dalla FDA per SKYTROFA® (lonapegsomatropin-tcgd) nel trattamento della carenza di ormone della crescita negli adulti (GHD). Questo rappresenta un'importante espansione rispetto all'approvazione pediatrica del GHD del 2021. SKYTROFA viene somministrato una volta alla settimana, offrendo un'alternativa meno gravosa rispetto alle tradizionali iniezioni giornaliere di somatropina.
L'approvazione si basa sullo studio fase 3 foresiGHt, che ha dimostrato efficacia e sicurezza rispetto al placebo e alla somatropina giornaliera. Questo traguardo è in linea con l'obiettivo Vision 2030 di Ascendis, che punta a diventare l'azienda leader nelle malattie endocrine rare, con piani per avviare ulteriori studi su diverse condizioni nel quarto trimestre del 2025.
Ascendis Pharma (Nasdaq: ASND) ha recibido la aprobación de la FDA para SKYTROFA® (lonapegsomatropin-tcgd) para el tratamiento de la deficiencia de hormona de crecimiento en adultos (GHD). Esto representa una expansión significativa desde su aprobación pediátrica de GHD en 2021. SKYTROFA se administra una vez a la semana, ofreciendo una alternativa menos exigente a las inyecciones diarias tradicionales de somatropina.
La aprobación se basó en el ensayo de fase 3 foresiGHt, que demostró eficacia y seguridad en comparación con placebo y somatropina diaria. Este hito está alineado con el objetivo Vision 2030 de Ascendis de convertirse en la empresa líder en enfermedades endocrinas raras, con planes para iniciar ensayos adicionales para diversas condiciones en el cuarto trimestre de 2025.
Ascendis Pharma (나스닥: ASND)는 성인 성장호르몬 결핍증(GHD) 치료를 위한 SKYTROFA®(로나펩소마트로핀-tcgd)의 FDA 승인을 받았습니다. 이는 2021년 소아 GHD 승인에서 크게 확장된 것입니다. SKYTROFA는 주 1회 투여로 기존의 매일 소마트로핀 주사보다 부담이 적은 대안을 제공합니다.
이번 승인은 위약 및 매일 소마트로핀과 비교하여 효능과 안전성을 입증한 foresiGHt 3상 시험을 기반으로 합니다. 이 성과는 Ascendis의 Vision 2030 목표인 희귀 내분비 질환 분야 선도 기업이 되겠다는 계획과 부합하며, 2025년 4분기에 다양한 질환에 대한 추가 임상시험을 시작할 예정입니다.
Ascendis Pharma (Nasdaq : ASND) a obtenu l'approbation de la FDA pour SKYTROFA® (lonapegsomatropin-tcgd) dans le traitement du déficit en hormone de croissance chez l'adulte (GHD). Cela représente une extension significative par rapport à son approbation pédiatrique du GHD en 2021. SKYTROFA est administré une fois par semaine, offrant une alternative moins contraignante aux injections quotidiennes traditionnelles de somatropine.
L'approbation s'appuie sur l'essai de phase 3 foresiGHt, qui a démontré l'efficacité et la sécurité par rapport au placebo et à la somatropine quotidienne. Cette étape s'inscrit dans l'objectif Vision 2030 d'Ascendis, visant à devenir l'entreprise leader dans les maladies endocriniennes rares, avec des plans pour lancer des essais supplémentaires pour diverses conditions au quatrième trimestre 2025.
Ascendis Pharma (Nasdaq: ASND) hat die FDA-Zulassung für SKYTROFA® (Lonapegsomatropin-tcgd) zur Behandlung von Wachstumshormonmangel bei Erwachsenen (GHD) erhalten. Dies stellt eine bedeutende Erweiterung gegenüber der pädiatrischen GHD-Zulassung aus dem Jahr 2021 dar. SKYTROFA wird einmal wöchentlich verabreicht und bietet eine weniger belastende Alternative zu den herkömmlichen täglichen Somatropin-Injektionen.
Die Zulassung basiert auf der foresiGHt Phase-3-Studie, die Wirksamkeit und Sicherheit im Vergleich zu Placebo und täglichem Somatropin nachwies. Dieser Meilenstein entspricht Ascendis' Vision 2030, führendes Unternehmen im Bereich seltener endokrinologischer Erkrankungen zu werden, mit Plänen, im vierten Quartal 2025 weitere Studien für verschiedene Erkrankungen zu starten.
- FDA approval expands SKYTROFA's market to adult GHD patients
- Once-weekly dosing provides competitive advantage over daily treatments
- Strong market position as 'treatment of choice' in pediatric GHD
- Pipeline expansion planned with multiple new indications in Q4 2025
- Multiple serious potential side effects including cancer risk and diabetes
- Complex safety profile requiring careful monitoring
- Significant contraindications limiting eligible patient population
Insights
FDA approval of SKYTROFA for adult GHD expands Ascendis' market reach with its once-weekly growth hormone therapy.
The FDA approval of SKYTROFA for adult growth hormone deficiency (GHD) represents a significant expansion of Ascendis Pharma's endocrinology portfolio. This approval builds upon the drug's initial 2021 authorization for pediatric GHD, creating a continuous treatment pathway across age groups.
The approval addresses a critical market need in adult GHD treatment. Current standard-of-care requires daily injections of somatropin, which creates adherence challenges. SKYTROFA's once-weekly administration offers a compelling competitive advantage that could drive market share gains in this indication.
This milestone aligns with Ascendis' strategic "Vision 2030" to become the leading endocrinology rare disease company. The company has clearly signaled additional expansion plans with an upcoming basket trial for multiple indications (ISS, SHOX deficiency, Turner syndrome, and SGA) and combination therapy trials in achondroplasia and hypochondroplasia planned for Q4 2025.
The approval was supported by the foresiGHt Phase 3 trial, which demonstrated efficacy against both placebo and active control (daily somatropin). Adult GHD is characterized by abnormal body composition, metabolic abnormalities, and quality of life impairments, creating a substantial medical need that SKYTROFA can now address with its unique sustained-release mechanism of unmodified somatropin.
This label expansion positions Ascendis to leverage its existing commercial infrastructure while potentially capturing increased market share in the growth hormone therapy space, particularly given the company's claim that SKYTROFA is already "the treatment of choice for pediatric GHD among patients and physicians."
- First of many planned label expansions supporting Vision 2030 goal to become the leading endocrinology rare disease company
- On track to initiate basket trial for ISS, SHOX deficiency, Turner syndrome, and SGA*, as well as combination therapy trials in achondroplasia and hypochondroplasia, in Q4 2025
COPENHAGEN, Denmark, July 28, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA® (lonapegsomatropin-tcgd; developed as TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD), a rare disorder resulting from decreased or total loss of growth hormone production. Lonapegsomatropin (approved by the FDA in 2021 for the treatment of pediatric GHD) is a prodrug of somatropin (human growth hormone, or hGH) administered once weekly, providing sustained release of active, unmodified somatropin.
The FDA’s approval of SKYTROFA for adult GHD was based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) clinical trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD.
“The abnormal body composition, dyslipidemia, and insulin resistance that are the hallmarks of adult GHD predispose affected individuals to serious medical complications such as metabolic syndrome, increased cardiovascular risk, and impaired quality of life – including cognitive dysfunction, depression, anxiety, sleep disturbance, and reduced physical and mental drive. Despite this, adherence to daily somatropin injections that have been the standard of care for more than 25 years remains a problem,” said Kevin Yuen, M.D., Barrow Neurological Institute, University of Arizona College of Medicine and Creighton University School of Medicine in Phoenix, Arizona. “It is no surprise that patients are looking for a less burdensome treatment regimen, and a new treatment option such as SKYTROFA, with once-weekly injection and its unique release of unmodified somatropin, is expected to help improve both real-world adherence and overall outcomes.”
“Our market research shows SKYTROFA is the treatment of choice for pediatric GHD among patients and physicians, and we are pleased to expand its availability in the United States for the treatment of adults initiating therapy or switching from another growth hormone therapy,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “This important milestone is the first of many planned label expansions supporting our goal to become the leading endocrinology rare disease company.”
The following information is intended for the U.S. Audience Only:
IMPORTANT SAFETY INFORMATION & USES
SKYTROFA is a prescription medicine used for:
- the replacement of growth hormone in children 1 year old or older who weigh at least 26
pounds (11.5 kilograms) with growth hormone deficiency (GHD) - the replacement of growth hormone in adults with growth hormone deficiency (GHD)
Do not take SKYTROFA if:
- you have a critical illness caused by certain types of heart or stomach surgery, trauma or breathing problems
- you are allergic to somatropin or any of the ingredients in SKYTROFA
- you are a child with closed bone growth plates
- you have cancer or other tumors
- you have certain types of eye problems caused by diabetes
- you are a child with Prader-Willi syndrome who is severely obese or has breathing problems including sleep apnea (briefly stopping breathing during sleep)
Tell your healthcare provider if you are pregnant or plan to become pregnant, about all of your medical conditions, and about all the medicines you take. SKYTROFA may affect how other medicines work, and other medicines may affect how SKYTROFA works.
What are the possible side effects of SKYTROFA?
SKYTROFA may cause serious side effects, including:
- serious allergic reactions. Get medical help right away if you have swelling of the face, mouth, tongue, or throat or trouble breathing
- high risk of death in people who have critical illnesses caused by heart or stomach surgery, trauma, or serious breathing problems
- increased risk of growth of an existing tumor or cancer, or increased risk of a previous tumor or cancer returning in people who were treated with radiation to the brain or head as children and who developed low growth hormone problems
- Your healthcare provider will need to monitor for the growth or return of a tumor or cancer. Contact your healthcare provider if you start to have sudden changes in behavior, headaches, vision problems, or changes in moles, birthmarks, or skin color
- new or worsening high blood sugar or diabetes. Your blood sugar may need to be monitored during treatment with SKYTROFA
- increase in pressure in the skull. Contact your healthcare provider if headaches, eye problems, nausea, or vomiting occur
- too much fluid in the body (fluid retention). Contact your healthcare provider if you notice swelling in the hands and feet, pain in the joints or muscles, or nerve problems that cause pain, burning or tingling in the hands, arms, legs, and feet
- decrease in a hormone called cortisol. Your healthcare provider will do blood tests to check your cortisol levels
- decrease in thyroid hormone levels. Decreased thyroid hormone levels may affect how well SKYTROFA works. Your healthcare provider will do blood tests to check your thyroid hormone levels
- hip and knee pain or a limp in children (slipped capital femoral epiphysis). This may lead to a serious condition where bone tissue dies due to a lack of blood supply (osteonecrosis). Get medical help right away for children who develop a limp or have hip or knee pain
- worsening of curvature of the spine in children (scoliosis)
- severe and constant abdominal pain. This could be a sign of pancreatitis. Tell your healthcare provider about any new abdominal pain
- loss of fat and tissue weakness in the area of skin you or your child inject. Talk to your healthcare provider about rotating the areas where SKYTROFA is injected
- high risk of sudden death in children with Prader-Willi syndrome who are severely obese or have breathing problems, including sleep apnea
- increase in phosphate, alkaline phosphatase, and parathyroid hormone levels in your blood. Your healthcare provider will do blood tests to check this
The most common side effects of SKYTROFA in children include: viral infection, fever, cough, nausea and vomiting, bleeding, diarrhea, stomach area pain, joint pain and arthritis
The most common side effects of SKYTROFA in adults include: swelling due to fluid build-up and low thyroid hormone
These are not all of the possible side effects of SKYTROFA. Call your doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch. You may also report side effects to Ascendis Pharma at 1-844-442-7236.
Please click here for SKYTROFA full Prescribing Information.
About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ plans to initiate basket trial for ISS, SHOX deficiency, Turner syndrome, and SGA, as well as combination therapy trials in achondroplasia and hypochondroplasia, in Q4 2025, (ii) SKYTROFA’s ability to improve real-world adherence and overall outcomes, (iii) Ascendis’ planned label expansions for SKYTROFA, (iv) Ascendis’ goal of becoming the leading endocrinology rare disease company, (v) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (vi) Ascendis’ application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA® are trademarks owned by the Ascendis Pharma group. © July 2025 Ascendis Pharma A/S.
| Investor Contacts: Sarada Weerasinghe Ascendis Pharma ir@ascendispharma.com | Media Contact: Melinda Baker Ascendis Pharma media@ascendispharma.com |
| Patti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com | |
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* ISS = idiopathic short stature
SHOX deficiency = short stature homeobox-containing gene deficiency
SGA = small for gestational age
FAQ
What is the FDA's new approval for SKYTROFA (ASND)?
How is SKYTROFA different from existing GHD treatments?
What were the results of SKYTROFA's Phase 3 foresiGHt trial?
What are the main side effects of SKYTROFA treatment?
What are Ascendis Pharma's future plans for SKYTROFA?
